Epidermolysis Bullosa (RDEB)
Topical QR-313 in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Due to Mutation(s) in Exon 73 of the COL7A1gene (WINGS)
A First in Human, Double-blind, Randomized, Intra-subject Placebo-controlled, Multiple Dose Study of QR-313 Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With RDEB Due to Mutation(s) in Exon 73 of the COL7A1 Gene
Phase 1/2
DESCRIPTION:
Four medical centers in CA, CO, MN, and OH are recruiting participants who have specific types of Recessive Dystrophic Epidermolysis Bullosa to test a new, topical treatment. The treatment aims to slow progression of EB wounds through a drug that binds to and stops the cellular messaging system that causes inflammation.
The actual drug and a placebo will be applied 2 to 3x per week to two wound areas, as assigned by the study, for 4 weeks. Home and site visits will follow for regular assessments up to 8 weeks post the last dose.
PATIENT MUST:
- Be 6 years of age or older
- Have at least 1 target wound area
- Have life expectancy > 6 months
THE STUDY INVOLVES:
- Screening before the treatment.
- Visit to the study center for assignment of the drug and/or placebo to the targeted wounds.
- For 4 weeks, the medicines will be applied to the assigned wounds 2 to 3 times per week.
- Home and site visits will follow for regular assessments up to 8 weeks post the last dose.
LOCATIONS AND CONTACTS:
The study locations include:
- Stanford University School of Medicine, LPCH in Palo Alto, CA
Contact: Kunju Sridhar | 650-721-4902 | [email protected] - Children’s Hospital Colorado in Aurora, CO
Contact: Kathleen Peoples | 720-777-4708 | [email protected] - Journey Clinic, Center for Pediatric Blood and Marrow Transplantation in Minneapolis, MN
Contact: Christen Ebens, MD | 612-626-8094 | [email protected] - Cincinnati Children’s Hospital in Cincinnati, OH
Contact: Bret Augsburger | 513-803-9009 | [email protected]
SPONSOR INFORMATION:
ProQR Therapeutics
Or go online: https://clinicaltrials.gov/ct2/show/NCT03605069